Natural history of acid sphingomyelinase deficiency (ASMD) among European patients during childhood and adolescence: A retrospective observational study

Mengel, Eugen; Scarpa, Maurizio; Guffon, Nathalie; Jones, Simon A.; Goriya, Vishal; Msihid, Jérôme; Dyevre, Valérie; Rodriguez, Carly A; Gašparić, Maja; Nalysnyk, Lubomyra; Laredo, Fernando; Pulikottil-Jacob, Ruth

doi:10.1016/j.ymgme.2022.107224

Molecular Genetics and Metabolism

2023

DOI: 10.1016/j.ymgme.2022.107224

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Natural history of acid sphingomyelinase deficiency (ASMD) among European patients during childhood and adolescence: A retrospective observational study

Eugen Mengel¹,

Maurizio Scarpa²,

Nathalie Guffon³

et al.

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Natural history of acid sphingomyelinase deficiency (ASMD) among European patients during childhood and adolescence: A retrospective observational study

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